Green light in Italy for the first and only therapy against a rare and aggressive type of blood cancer. The Italian Medicines Agency (AIFA) has approved the reimbursement of tagraxofusp as monotherapy for the first-line treatment of patients with blastic plasmacytoid dendritic cell neoplasia (BPDCN), a rare blood cancer with a severe prognosis characterized by hyper- CD123 antigen expression. This is the first and only specific therapy for this pathology, the result of research and development by Menarini Stemline.
Italy is the second country in Europe to make the drug available, after Germany. Aifa’s provision follows approval at the European level.
“Before tagraxofusp, there were no authorized drugs in this neoplasm and, in clinical practice, intensive chemotherapy regimens were normally used for the treatment of leukemia or lymphomas – says Pier Luigi Zinzani, Full Professor of the Institute of Hematology ‘LA
Seràgnoli’, IRCCS S. Orsola-Malpighi, University of Bologna -. Blastic plasmacytoid dendritic cell neoplasm, however, is characterized by an intrinsic resistance to standard chemotherapy: the responses are often transient and the results obtained do not translate into advantages from the point of view of survival. These chemotherapy regimens are also associated with severe toxicities.” Tagraxofusp is instead a targeted, ‘first-in-class’ therapy that acts selectively against the CD123 antigen. In the pivotal study involving 89 patients, the drug resulted in an overall response of 75% and disease remission was 57%.51% of patients in remission then underwent successful hematopoietic stem cell transplantation. Tagraxofusp “is able to bring the tumor into remission, which can then be consolidated with hematopoietic stem cell transplantation, thus improving the chances of recovery. The only treatment capable of achieving this important result is the transplant, but not all patients can undergo it – explains Emanuele Angelucci, Director of the Complex Structure of Hematology and Cellular Therapies of the IRCCS San Martino Polyclinic Hospital in Genoa -. Tagraxofusp may increase the number of patients who achieve remission without subjecting them to the toxicity of intensive chemotherapy and who, therefore, are candidates for transplantation.”
The disease, which usually presents at the onset with multiple, infiltrating dark skin lesions, in its course can affect the bone marrow, central nervous system and lymph nodes, thus becoming a systemic pathology.
It has a very low incidence, there are less than one hundred new cases every year in Italy, and this neoplasm has only recently found an adequate definition. Today it is classified under aggressive myeloid malignancies.
In August 2019, the manufacturer launched an Expanded Access Program (EAP) in Europe to guarantee patients access to tagraxofusp before its approval by the EMA and, at the same time, collect data on its efficacy and safety in daily clinical practice. “The drug – continues Angelucci – also showed a favorable risk-benefit profile in ‘real life’, with an efficacy that appears even higher than that reported in the pivotal trial. The preliminary results of the European retrospective study ‘Expanded Access Program (EAP )’, in which Italy also participated, were presented last December at the congress of the American Society of Hematology.The results of a preliminary analysis of the data of 22 patients treated with tagraxofusp were reported, of which 15 on the first line “In these, the overall response was 87% and the complete remission rate was 67%. 50% underwent stem cell transplantation. These are extremely satisfactory data, given the aggressiveness of the disease”. .
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